Myelodysplastic syndromes. Progress in diagnosis and treatment within 30 years of myelodysplastic syndromes registry in Institute of Hematology in Prague

Česká verzia

Authors: J. Čermák
Authors place of work: Ústav hematologie a krevní transfuze Praha, ředitel prof. MUDr. Marek Trněný, CSc.
Published in the journal: Vnitř Lék 2012; 58(Suppl 2): 8-15
Category:

Summary

An analysis of data obtained from 367 patients with primary myelodysplastic syndromes (MDS) treated in Institute of Hematology and Blood Transfusion in the years 1980–2009 revealed substantional changes in diagnostic and therapeutic approaches within past 30 years. Molecular biology methods have been recently incorporated into the set of diagnostic methods and the importance of results obtained by molecular genetic methods for prognosis and optimal treatment is intensively studied. The treatment approaches switched from supportive care and palliative chemotherapy to new drugs that may effectively target the molecular basis of the disease as lenalidomide in early disease and hypomethylating agents in advanced MDS. Introduction of stem cell transplantation (SCT) significantly improved outcome especially in younger patients with advanced MDS and SCT still represents the only curative treatment approach.

Key words:
myelodysplastic syndromes – diagnosis – treatment – prognosis – registry

Zdroje

1. Björkman SE. Chronic refractory anemia with sideroblastic bone marrow: a study of four cases. Blood 1956; 11: 250–259.

2. Bennett J, Catovsky D, Daniel MT et al. Proposals for the classification of the myelodysplastic syndromes. Br J Haematol 1982; 51: 189–199.

3. Raza A, Gezer S, Mundle S et al. Apoptosis in bone marrow biopsy samples involving stromal and hematopoietic cells in 50 patients with myelodysplastic syndromes. Blood 1995; 86: 268–276.

4. Parker JE, Mufti GJ. Ineffective haemopoiesis and apoptosis in myelodysplastic syndromes. Br J Haematol 1998; 101: 220–230.

5. Brunning RD, Orazi A, Germing U et al. Myelodysplastic syndromes/neoplasms, overview. In: Swerdlow SH, Campo E, Harris NL et al (eds). WHO Classification of Tumours of Haematopoietic nad Lymphoid Tissues. 4th ed. Lyon: IARC Press 2008: 88–93.

6. Greenberg P, Cox C, LeBeau MM et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 1997; 89: 2079–2088.

7. Schanz J, Tüchler H, Solé F et al. A new comprehensive cytogenetic scoring system for primary myelodysplastic syndromes (MDS) and oligoblastic AML after MDS derived from an international database merge. J Clin Oncol 2012; 30: 820–829.

8. Tiu RV, Gondek LP, O’Keefe CL et al. Prognostic impact of SNP array karyotyping in myelodysplastic syndromes and related myeloid malignancies. Blood 2011; 117: 4552–4560.

9. van de Loosdrecht AA, Alhan C, Béné MC et al. Standardization of flow cytometry in myelodysplastic syndromes: report from the first European LeukemiaNet working conference on flow cytometry in myelodysplastic syndromes. Haematologica 2009; 94: 1124–1134.

10. Májek P, Reicheltová Z, Suttnar J et al. Plasma proteome changes associated with refractory cytopenia with multilineage dysplasia. Proteome Sci 2011; 9: 64.

11. Sanz GF, Sanz MA, Vallespí T et al. Two regression models and a scoring system for predicting survival and planning treatment in myelodysplastic syndromes: a multivariate analysis of prognostic factors in 370 patients. Blood 1989; 74: 395–408.

12. Harris NL, Jaffe ES, Diebold J et al. World Health Organization classification of neoplastic diseases of the hematopoietic and lymphoid tissues: report of the Clinical Advisory Committee meeting – Airlie House, Virginia, November 1997. J Clin Oncol 1999; 17: 3835–3849.

13. Malcovati L, Germing U, Kuendgen A et al. Time-dependent prognostic scoring system for predicting survival and leukemic evolution in myelodysplastic syndromes. J Clin Oncol 2007; 25: 3503–3510.

14. Čermák J, Kacirkova P, Mikulenkova D et al. Impact of transfusion dependency on survival in patients with early myelodysplastic syndrome without excess of blasts. Leuk Res 2009; 33: 1469–1474.

15. Dostálová Merkerová M, Bystrická D, Belicková M et al. From cryptic chromosomal lesions to pathologically relevant genes: integration of SNP-array with gene expression profiling in myelodysplastic syndrome with normal karyotype. Genes Chromosomes Cancer 2012; 51: 419–428.

16. Vasikova A, Belickova M, Budinska E et al. A distinct expression of various gene subsets in CD34+ cells from patients with early and advanced myelodysplastic syndrome. Leuk Res 2010; 34: 1566–1572.

17. Bejar R, Stevenson K, Abdel-Wahab O et al. Clinical effect of point mutations in myelodysplastic syndromes. N Eng J Med 2011; 364: 2496–2506.

18. Malcovati L, Papaemmanuil E, Bowen DT et al. Clinical significance of SFR3Bl mutations in myelodysplastic syndromes and myelodysplastic/myeloproliferative neoplasms. Blood 2011; 118: 6239–6246.

19. Bennett JM. Consensus statement on iron overload in myelodysplastic syndromes. Am J Hematol 2008; 83: 858–861.

20. Hellström-Lindberg E. Efficacy of erythropoietin in the myelodysplastic syndromes: a meta-analysis of 205 patients from 17 studies. Br J Haematol 1995; 89: 67–71.

21. Passweg JR, Giagounidis AA, Simcock M et al. Immunosuppressive therapy for patients with myelodysplastic syndrome: a prospective randomized multicenter phase III trial comparing antithymocyte globulin plus cyclosporine with best supportive care – SAKK 33/99. J Clin Oncol 2010; 29: 303–309.

22. Sloand EM, Wu CO, Greenberg P et al. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol 2008; 26: 2505–2511.

23. Bejar R, Levine R, Eberet BJ. Unraveling the molecular pathophysiology of myelodysplastic syndromes. J Clin Oncol 2011; 29: 504–515.

24. Kuendgen A, Lauseker M, List AM et al. Lenalidomid treatment is not related to AMl progression risk but is associated with survival benefit in RBC-dependent transfusion patients with IPSS Low- or Int1-Risk MDS with del5q. Blood 2011; 118. Abstract 119.

25. Cermák J, Vítek A, Michalová K. Combined stratification of refractory anemia according to both WHO and IPSS criteria has a prognostic impact and improves identification of patients who may benefit from stem cell transplantation. Leuk Res 2004; 28: 551–557.

26. Cutler CS, Lee SJ, Greenberg P et al. A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood 2004; 104: 579–585.

27. Fenaux P, Mufti GJ, Hellstrom-Lindberg E et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk MDS: a randomized, open-label, phase III study. Lancet Oncology 2009; 10: 223–232.

28. Martino R, Iacobelli S, Brand R et al. Retrospective comparison of reduced-intensity conditioning and conventional high-dose conditioning for allogeneic hematopoietic stem cell transplantation using HLA-identical sibling donors in myelodysplastic syndromes. Blood 2006; 108: 836–846.

29. Schmid C, Schleuning M, Ledderose G et al. Sequential regimen of chemotherapy, reduced-intensity conditioning for allogeneic stem-cell transplantation, and prophylactic donor lymphocyte transfusion in high-risk acute myeloid leukemia and myelodysplastic syndrome. J Clin Oncol 2005; 23: 5675–5687.

30. Cermak J, Vitek A, Markova M et al. Combination chemotherapy leading in advanced MDS patients in a rapid clearence of bone marrow blasts prior stem cell transplantation (SCT) is superior to up-front SCT even with intensified conditiong for long-term survival. Blood 2010; 116. Abstract 4020.